|Report Code: PP10125||Report Type: Indication Pipeline Reports||Available format:|
Urea cycle disorders (UCDs) are an inborn error of metabolism, that occurs due to the deficiencies in any of the six enzymes and two transport proteins involved in urea biosynthesis. These disorders cause inborn errors in urea synthesis that lead to an accumulation of ammonia in blood and brain. UCDs clinically present as recurrent episodes of hyperammonemia manifested by vomiting, lethargy and coma. The diagnosis of UCDs occurs during neonatal screening; while, some milder cases can diagnose later. The management of UCDs is based on minimizing the nitrogen load by decreasing dietary protein intake. Thus, patients generally consume a protein-restricted diet in order to minimize the production of ammonia from dietary nitrogen.
The drug candidates in UCDs therapeutics pipeline include, but not limited to, SYNB1020 and mRNA therapeutics. Some of the major companies having pipeline drugs for UCDs includes PhaseRx Inc., Synlogic and Castle Creek Pharma.